FDA chief: Drug approval balance of risk, benefits

Feb. 26, 2014 @ 05:33 PM

Drugs that come off the market are not a sign of a failure of the regulatory system, the head of the U.S. Food and Drug Administration said at a life sciences conference in Raleigh on Wednesday.
FDA Commissioner Dr. Margaret Hamburg spoke at the annual conference hosted by Durham-based CED, the nonprofit entrepreneurial support organization formally known as the Center for Entrepreneurial Development. The first day of the conference drew drug development leaders, researchers, and others to the Raleigh Convention Center. The conference was expected to draw hundreds of people.
“Drugs coming off market (are not) a failure,” Hamburg said, explaining that the agency has evolved over time from a view that it must protect patients to a view that it must protect patients using research “in order to ensure access to new and effective medicines.”
Each drug that is brought to the market always involves a balance of risks and benefits, she said. There are side effects and problems that may emerge after a drug is more widely used by the public. The agency has to balance the risk with the “eagerness to address the needs of patients with the most serious of illnesses.”
Also on Wednesday, Hamburg described agency regulatory process changes that she said are exciting, but not without controversy. She described pathways the agency has in place to fast-track approval of certain drugs, such as breakthrough therapy designation created by Congress in 2012. Forty new drugs have been designated for review under that track, she said, and three have been approved.
Last year overall, she said the agency’s Center for Drug Evaluation and Research approved 27 new medicines, of which “a very large number” were approved using one or in some cases, multiple expedited approval pathways.
While she said that news is “exciting,” she also said the agency does have its critics who think the organization is sacrificing safety standards for speed. But she said regulatory safety doesn’t mean safety standards are relaxed, and said she believes that a “one-size fits all” pathway for all new medicines is “simply not the answer.”
“Increased flexibility does not mean abandoning standards and it certainly does not mean abandoning science,” she said.
Also, Hamburg described areas of emerging medical research ranging work into the use of a patient’s own stem cells to grow missing or damaged tissue to the use of genetics to tailor medications to individual patients, to the use of 3-D printing for making new body parts for patients using scans or other imaging technologies.
“Three-(dimensional) printing has a growing place in medicine,” Hamburg said. “For example, it allows complex and moving parts to be created for use in the body, and more importantly, to be designed from patient imaging…,” she added.
Mobile applications were another emerging area that Hamburg said have the potential to provide “enormous benefits to consumers” related to their health. Some apps can help people maintain their weight, she said, while others can help diabetics check their blood-sugar levels. Guidance released by the agency last year focused on a limited group of mobile apps considered to have the greatest risk, she said.
“(That’s) why recently (we) put out a guidance to cover the field of emerging technology (to) promote innovation while protecting patients,” she said.
According to a news release from the FDA from September of last year about the new guidance, the agency is focusing regulation on apps intended to be used as an accessory to a regulated medical device, or that transform a mobile device into a medical device. The release said the apps that go through the agency’s review will be assessed using the same regulatory standards that apply to other medical devices.
There are mobile medical apps on the market that can diagnose abnormal heart rhythms and transform smart phones into mobile ultrasound devices, the release said.
Along with Hamburg’s speech, the conference also included panels of discussions between executives at drug companies and as well as at companies that work with them. There was an innovation showcase dedicated to showing what emerging companies are working on in North Carolina, in including one company that’s working on a blood test to aid in detection of breast cancer.
Dr. William Roper, dean of the School of Medicine, vice chancellor for Medical Affairs at the University of North Carolina at Chapel Hill and CEO of the UNC Health Care system, also attended part of the conference Wednesday. In a separate interview, Roper described what he believes is a “major shift” in the way pharmaceutical research and manufacturing companies are operating. He said they’re choosing to downsize their internal operations and to partner with universities to do “much of their research.”
“There’s a real opportunity for higher education and the pharmaceutical industry to partner,” Roper said. “I’m very bullish on that…”
While he said the university will continue to look forward to research funding from organizations like the National Institutes of Health, he said he also expects more funding to come from the pharmaceutical industry. He said that presents challenges, including that there are some people who are “made fearful by the notion of the university doing more with the private sector.”
“But happily, I think it can be worked out,” Roper said.